Pa­tient-fo­cused drug de­vel­op­ment: New FDA draft guid­ance delves in­to what’s im­por­tant

The FDA on Mon­day pub­lished the sec­ond in a se­ries of four guid­ance doc­u­ments on pa­tient-fo­cused drug de­vel­op­ment, with this lat­est draft help­ing spon­sors iden­ti­fy what is most im­por­tant to pa­tients with re­spect to their ex­pe­ri­ence with dis­ease bur­den and treat­ment.

In de­ter­min­ing what’s im­por­tant, the FDA rec­om­mends that spon­sors use qual­i­ta­tive re­search meth­ods, which can gen­er­ate in-depth thoughts from pa­tients in their own words through in­ter­views or fo­cus groups, quan­ti­ta­tive re­search meth­ods, which can use sta­tis­ti­cal meth­ods to sum­ma­rize the col­lec­tive pa­tient ex­pe­ri­ence da­ta, or mixed-meth­ods re­search, which can com­bine qual­i­ta­tive and quan­ti­ta­tive meth­ods to un­der­stand the pa­tient ex­pe­ri­ence.

The 45-page draft dis­cuss­es sev­er­al ways to col­lect qual­i­ta­tive in­for­ma­tion, in­clud­ing via one-on-one in­ter­views and fo­cus groups, with hints on how to ask the right types of ques­tions.

On quan­ti­ta­tive meth­ods, the draft ex­plains dif­fer­ent sur­veys and what to con­sid­er, in­clud­ing ex­am­ples of what could po­ten­tial­ly be lead­ing or dou­ble-neg­a­tive ques­tions.

Ex­am­ples of mixed-method study de­signs are al­so ex­plained fur­ther, along­side ques­tions re­searchers should ask them­selves and rea­sons to use a mixed-method de­sign.

In ad­di­tion, the draft in­cludes a sec­tion on spe­cif­ic pop­u­la­tions, cul­tur­al­ly di­verse pop­u­la­tions and con­sid­er­a­tions for us­ing so­cial me­dia, which dis­cuss­es the im­por­tance of keep­ing the var­i­ous so­cial plat­forms and users in mind.

“When pos­si­ble, so­cial me­dia re­search should ex­am­ine a va­ri­ety of so­cial me­dia net­works and com­mu­ni­ties to ob­tain da­ta that can be most gen­er­al­ized to the pop­u­la­tion of in­ter­est,” the agency adds. The draft al­so in­cludes six ap­pen­dices, in­clud­ing a glos­sary of terms and dif­fer­ent types of ex­am­ples for each of the re­search meth­ods.

The re­lease of the draft guid­ance co­in­cides with the Mon­day re­lease of a new per­spec­tive ar­ti­cle on en­gag­ing pa­tients co-au­thored by both FDA and EMA of­fi­cials in Na­ture Re­views Drug Dis­cov­ery.

The per­spec­tive fo­cus­es on ini­tia­tives from both reg­u­la­tors that have al­lowed for fur­ther pa­tient or pub­lic en­gage­ment, and fur­ther en­gage­ment be­tween them.

“Our re­spon­si­bil­i­ty is the same for all pa­tients: to en­sure that they re­ceive the safest, most ef­fec­tive med­i­cines and that, as much as pos­si­ble, their needs and con­cerns are con­sid­ered in the eval­u­a­tion process,” EMA and FDA of­fi­cials wrote. “Giv­en the glob­al as­pects of med­i­cine reg­u­la­tion, col­lab­o­rat­ing and shar­ing ex­pe­ri­ences, in­for­ma­tion and re­sources be­tween agen­cies is al­so vi­tal, and ex­changes such as those seen through the col­lab­o­ra­tion be­tween the EMA and the FDA are help­ing to im­prove pa­tients’ trust in and un­der­stand­ing of the reg­u­la­to­ry process.”

The FDA, mean­while, which pre­vi­ous­ly re­leased the first pa­tient-fo­cused drug de­vel­op­ment draft guid­ance in June 2018, un­der this new ini­tia­tive cre­at­ed by the 21st Cen­tu­ry Cures Act, will al­so re­lease two oth­er guid­ance doc­u­ments deal­ing with ap­proach­es to iden­ti­fy and de­vel­op meth­ods to mea­sure im­pacts in clin­i­cal tri­als and meth­ods, stan­dards and tech­nolo­gies to col­lect and an­a­lyze clin­i­cal out­come as­sess­ment da­ta for reg­u­la­to­ry de­ci­sion-mak­ing.

Pa­tient-Fo­cused Drug De­vel­op­ment: Meth­ods to Iden­ti­fy What Is Im­por­tant to Pa­tients Guid­ance for In­dus­try, Food and Drug Ad­min­is­tra­tion Staff, and Oth­er Stake­hold­ers


RAPS: First pub­lished in Reg­u­la­to­ry Fo­cus™ by the Reg­u­la­to­ry Af­fairs Pro­fes­sion­als So­ci­ety, the largest glob­al or­ga­ni­za­tion of and for those in­volved with the reg­u­la­tion of health­care prod­ucts. Click here for more in­for­ma­tion.

Ab­b­Vie wins an ap­proval in uter­ine fi­broid-as­so­ci­at­ed heavy bleed­ing. Are ri­vals My­ovant and Ob­sE­va far be­hind?

Women expel on average about 2 to 3 tablespoons of blood during their time of the month. But with uterine fibroids, heavy bleeding is typical — a third of a cup or more. Drugmakers have been working on oral therapies to try and stem the flow, and as expected, AbbVie and their partners at Neurocrine Biosciences are the first to make it across the finish line.

Known chemically as elagolix, the drug is already approved as a treatment for endometriosis under the brand name Orilissa. It targets the GnRH receptor to decrease the production of estrogen and progesterone.

Sanofi brings in 4 new ex­ec­u­tives in con­tin­ued shake-up, as vac­cines and con­sumer health chief head out the door

In the middle of Sanofi’s multi-pronged race to develop a Covid-19 vaccine, David Loew, the head of their sprawling vaccines unit, is leaving – part of the final flurry of moves in the French giant’ months-long corporate shuffle that will give them new-look leadership under new CEO Paul Hudson.

The company also said today that Alan Main, the head of their consumer healthcare unit, is out, and they named 4 executives to fill new or newly vacated positions, 3 of whom come from both outside both Sanofi and from Pharma.

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As­traZeneca trum­pets the 'mo­men­tous' da­ta they found for Tagris­so in an ad­ju­vant set­ting for NSCLC — but many of the ex­perts aren’t cheer­ing along

AstraZeneca is rolling out the big guns this evening to provide a salute to their ADAURA data on Tagrisso at ASCO.

Cancer R&D chief José Baselga calls the disease-free survival data for their drug in an adjuvant setting of early stage, epidermal growth factor receptor-mutated NSCLC patients following surgery “momentous.” Roy Herbst, the principal investigator out of Yale, calls it “transformative.”

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Pablo Legorreta, founder and CEO of Royalty Pharma AG, speaks at the annual Milken Institute Global Conference in Beverly Hills, California (Patrick T. Fallon/Bloomberg via Getty Images)

Cap­i­tal­iz­ing Pablo: The world’s biggest drug roy­al­ty buy­er is go­ing pub­lic. And the low-key CEO di­vulges a few se­crets along the way

Pablo Legorreta is one of the most influential players in biopharma you likely never heard of.

Over the last 24 years, Legorreta’s Royalty Pharma group has become, by its own reckoning, the biggest buyer of drug royalties in the world. The CEO and founder has bought up a stake in a lengthy list of the world’s biggest drug franchises, spending $18 billion in the process — $2.2 billion last year alone. And he’s become one of the best-paid execs in the industry, reaping $28 million from the cash flow last year while reserving 20% of the cash flow, less expenses, for himself.

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Dan O'Day, Gilead CEO (Andrew Harnik, AP Images)

UP­DAT­ED: Gilead leas­es part­ner rights to TIG­IT, PD-1 in a $2B deal with Ar­cus. Now comes the hard part

Gilead CEO Dan O’Day has brokered his way to a PD-1 and lined up a front row seat in the TIGIT arena, inking a deal worth close to $2 billion to align the big biotech closely with Terry Rosen’s Arcus. And $375 million of that comes upfront, with cash for the buy-in plus equity, along with $400 million for R&D and $1.22 billion in reserve to cover opt-in payments and milestones..

Hotly rumored for weeks, the 2 players have formalized a 10-year alliance that starts with rights to the PD-1, zimberelimab. O’Day also has first dibs on TIGIT and 2 other leading programs, agreeing to an opt-in fee ranging from $200 million to $275 million on each. There’s $500 million in potential TIGIT milestones on US regulatory events — likely capped by an approval — if Gilead partners on it and the stars align on the data. And there’s another $150 million opt-in payments for the rest of the Arcus pipeline.

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Paul Hudson, Sanofi CEO (Getty Images)

Sanofi CEO Paul Hud­son has $23B burn­ing a hole in his pock­et. And here are some hints on how he plans to spend that

Sanofi has reaped $11.1 billion after selling off a big chunk of its Regeneron stock at $515 a share. And now everyone on the M&A side of the business is focused on how CEO Paul Hudson plans to spend it.

After getting stung in France for some awkward politicking — suggesting the US was in the front of the line for Sanofi’s vaccines given American financial support for their work, versus little help from European powers — Hudson now has the much more popular task of managing a major cash cache to pull off something in the order of a big bolt-on. Or two.

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Roger Perlmutter, Merck R&D chief (YouTube)

Backed by BAR­DA, Mer­ck jumps in­to Covid-19: buy­ing out a vac­cine, part­ner­ing on an­oth­er and adding an­tivi­ral to the mix

Merck execs are making a triple play in a sudden leap into the R&D campaign against Covid-19. And they have more BARDA cash backing them up on the move.

Tuesday morning the pharma giant simultaneously announced plans to buy an Austrian biotech that has been working on a preclinical vaccine candidate, added a collaboration on another vaccine with the nonprofit IAVI and inked a deal with Ridgeback Biotherapeutics on an early-stage antiviral.

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As­traZeneca’s $7B ADC suc­ceeds where Roche failed, im­prov­ing sur­vival in gas­tric can­cer

Another day, another win for Enhertu.

The antibody-drug conjugate AstraZeneca promised up-to $7 billion to partner on has had a quite a few months, beginning with splashy results in a Phase II breast cancer trial, a rapid approval and, earlier this month, breakthrough designations in both non-small cell lung cancer and gastric cancer.

Now, at ASCO, the British pharma and their Japanese partner, Daiichi Sankyo, have shown off the data that led to the gastric cancer designation, which they’ll take back to the FDA. In a pivotal, 187-person Phase II trial, Enhertu shrunk tumors in 42.9% of third-line patients with HER2-positive stomach cancer, compared with 12.5% in a control arm where doctors prescribed their choice of therapy. Progression-free survival was 5.4 months for Enhertu compared to 3.5 months for the control.

Once a gem, now just a rock, Take­da punts PhI­II IBD drug as ri­vals mus­cle ahead

Back in 2016, when then-Shire CEO Flemming Ørnskov picked up a promising clinical-stage IBD drug from Pfizer, the Boston-based biotech dubbed it SHP647 and moved it into the gem section of the pipeline, with rosy expectations of registration-worthy Phase III data ahead.

This was a drug that the EC wanted Takeda to commit to selling off before it gave their blessing to its acquisition of Shire, to settle some deep-seated concerns revolving around the potential market overlap with their blockbuster rival Entyvio. And Takeda, which took on a heavy debt load to buy Shire, clearly wanted the cash to pay down debt.